Get Ready for Comparative Effectiveness Research

May 2010, Vol 1, No 1 - ACCC Conference

Baltimore, MD—Comparative effectiveness research (CER) is at the forefront of new cost-control efforts, according to experts speaking at a panel discussion at the Asso ciation of Community Cancer Centers’ (ACCC) 36th Annual National Meeting. Unlike traditional drug evaluations conducted by the US Food and Drug Administration, CER aims to evaluate the clinical and cost-effectiveness of treatments through head-to-head trials. And although CER promises important new clinical insights, no one doubts that rising healthcare costs are forcing payers and providers alike to think about driving value.

Al B. Benson III, MD“Although you keep hearing this isn’t about money, I don’t think anyone believes that for 1 second,” said Al B. Benson III, MD, and incoming president of the ACCC. “It’s all about money. It’s going to affect our pocketbooks in many ways. We’re all going to see reimbursement based on supplying data.”

Exploding healthcare costs and the new healthcare law have focused increased attention on CER. The Recovery Act of 2009 dedicated $1.1 billion to CER, and the healthcare reform law creates a Patient-Centered Outcomes Research Institute to coordinate federal CER support, seeded with hundreds of millions of dollars in new funding.

“The purpose for setting it up…was to create an independent entity that would be separate from government pressure and have a sustained funding source,” Jeff Allen, PhD, executive director of Friends of Cancer Research, told Value-Based Cancer Care.

Advocates say the funds are just a start. In a recent perspective, Jerry Avorn, MD, wrote that, “at 1/20 of 1% of our $2-trillion annual healthcare expenditure, the CER funding amounts to a fraction of what any corporation would spend to find out whether it was getting its money’s worth from its purchases” (N Engl J Med. 2009;360[19]:1927-1929).

Given the intensely partisan environment in Washington, CER has become a political football of sorts: Republicans have charged that CER will lead to rationed care; and pharmaceutical companies and device makers eager to get their products to market are nervous about lost profits.

In an attempt to placate critics, the stimulus law included a provision ostensibly limiting the role CER could play in reimbursement, but the clause is more politics than substance: Dr Allen said that the Centers for Medicare & Medicaid Services will likely treat CER data the same way they treat any research in evaluating cost and clinical effectiveness.

Others worry that CER will undermine patient-based medicine, and some advocates worry it could gloss over differences among subpopulations. At the session, Dr Allen worried that the paltry information provided by the current model is a more significant concern, and said that CER should enhance the identifications of subset variations. Consumer advocate groups such as the Consumers Union have backed CER.

Problems and Priorities

A recent analysis of medication comparative effectiveness studies found that the majority of the studies examined did not compare drugs to currently available therapies, and that many simply measured drugs against a placebo (JAMA. 2010;303[10]:951-958). The authors blame the pharmaceutical industry for skewing research toward bringing new drugs to market. They also found that nonprofit foundations or the government funded most comparative effectiveness studies, so it seems likely that public funding will drive CER in the future.

In addition to funding, government is providing guidance on how CER should be pursued. In June 2009, the Institute of Medicine released 100 top priorities for CER, including 6 in the fields of oncology and hematology. These include comparing the effectiveness of genetic and biomarker testing for preventing and treating cancer; effectiveness of management strategies for localized prostate cancer; effectiveness of ductal carcinoma in situ management strategies; and the use of diagnostic imaging, an issue that has been in the news a lot and was on the mind of Dr Benson.

“We talk in oncology organizations a great deal about drug reimbursement,” Dr Benson noted. “Well, that pales when you look at imaging. And we in oncology order a lot of tests. We make a lot of money for our radiology department. And unfortunately, when you look at the literature, there’s very little guidance with how to use imaging.”

Beyond the politics of the situation, one of the biggest challenges will be the collection of data. “We do have to generate evidence,” said H. Kim Lyerly, MD. “One of the challenges, though, is cost.” Since hospitals already collect much of this data, electronic health records would not be necessary—but they would be a big help in making data collection more affordable.

“What we would hope is that this would be one component of practicing evidence-based medicine,” said Dr Benson.

There is no shortage of methodological questions to work out, including how to ensure that outliers are identified. And once that CER data gets generated, it will have to percolate down into the day-to-day of clinical practice.

“Getting new research findings into the hands of doctors and patients is easier said than done,” said Dr Allen. “Some estimate that it takes upwards of a decade to implement a new research finding into routine clinical practice.”